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FDA approval—first gene therapy under new priority voucher for hearing loss
Regeneron’s gene therapy Otarmeni (lunsotogene parvec) won U.S. FDA approval for a rare inherited form of hearing loss caused by variants in the otoferlin gene. The approval marks the first...
Gene editing—base editing corrects hard-to-treat CFTR splicing mutation in cell models
A new cell-model study reports functional correction of the “untreatable” cystic fibrosis mutation 1717-1G>A using CRISPR base editing. Researchers from the University of Trento and collaborators...
Dealmaking—Ray Therapeutics raises $125M to advance retinal gene therapy into registrational study
Ray Therapeutics closed a $125 million Series B financing to accelerate development of its optogenetic retinal gene therapy RTx-015 for retinitis pigmentosa. The funding is earmarked for...
Oncology—AACR early data spotlight new ADCs and targeted agents
At AACR 2026, multiple early-phase oncology readouts reinforced the sector’s continued focus on ADCs and targeted therapies in hard-to-treat populations. In separate presentations, researchers...
Immunotherapy in autoimmune disease—Kyverna’s miv-cel shows positive registrational Phase II primary analysis
Kyverna Therapeutics reported positive primary-analysis results from its registrational Phase II trial KYSA-8 of mivocabtagene autoleucel (miv-cel, KYV-101) in stiff-person syndrome. The company...
CGT and manufacturing—Andelyn and ENCell form U.S.-Asia manufacturing bridge for gene therapies
Andelyn Biosciences partnered with South Korea-based ENCell to build a dual-hemisphere manufacturing bridge aimed at accelerating global delivery of cell and gene therapies. The collaboration is...
Diagnostics and biosensing—Claret Bioscience and NEB collaborate on methylation workflow with NEBNext EM-seq chemistry
Claret Bioscience struck a copromotion deal with New England Biolabs to combine Claret’s SRSLY MethylPlus library prep approach with NEBNext EM-seq v2 Conversion Module chemistry for DNA...
Bioprocessing automation—Sartorius launches CellCelector CLD for high-throughput monoclonal cell line development
Sartorius unveiled the CellCelector CLD, an automated imaging and cell isolation platform intended to accelerate monoclonal cell line development. The system combines high-speed scanning,...
Policy and coverage—FDA and CMS create RAPID pathway to align breakthrough device review and Medicare coverage timelines
The U.S. FDA and Centers for Medicare & Medicaid Services announced a new regulatory alignment pathway, RAPID, designed to shorten the delay between FDA authorization of breakthrough medical...
Corporate and macro in pharma—biopharma M&A adds milestones and contingent payments
A new sector analysis highlights a shift in biopharma deal structures, with more large transactions incorporating contingent milestone payments and “CVR”-style mechanics. The report says...
FDA approval expands gene therapy access for hearing loss
The U.S. FDA approved Regeneron Pharmaceuticals’ gene therapy Otarmeni (lunsotogene parvec) for a rare, inherited form of hearing loss caused by otoferlin gene variants, marking the first gene...
Cell therapy pushes into autoimmune disease with miv-cel in stiff-person syndrome
Kyverna Therapeutics reported positive primary phase II results from its registrational trial KYSA-8 of mivocabtagene autoleucel (miv-cel, KYV-101) in stiff-person syndrome. In an oral...
Targeted oncology: RAS-focused medicines regain momentum at AACR
At AACR 2026, Revolution Medicines and related programs delivered additional clinical signals for RAS-directed approaches in pancreatic cancer, with updated data spanning monotherapy and...
New gene-editing strategy targets ‘untreatable’ CFTR splicing mutation
Researchers from the University of Trento and collaborators reported a base-editing approach designed to functionally correct the severe cystic fibrosis CFTR 1717-1G>A splicing mutation using...
Funding: Ray Therapeutics raises to advance retinal gene therapy toward registration
Ray Therapeutics closed a $125 million Series B financing to fund late-stage development and commercialization readiness for its lead optogenetic gene therapy RTx-015 in retinitis pigmentosa. Paul...
Biopharma dealmaking: Biogen buys rights in Greater China for felzartamab
Biogen entered a definitive agreement with TJ Biopharma to acquire exclusive Greater China rights to felzartamab, a CD38-directed monoclonal antibody in development across immune-mediated...
Diagnostics and interoperability: FDA and CMS align to speed Medicare coverage decisions for breakthrough devices
The FDA and CMS announced a new RAPID coverage pathway designed to reduce the lag between FDA authorization of breakthrough devices and Medicare national coverage decisions. Under the approach,...
CGT manufacturing: Sartorius launches automated clone selection platform for monoclonal cell line development
Sartorius introduced the CellCelector CLD, an automated imaging and cell isolation platform targeted at monoclonal cell line development. The system is designed to shorten the clone development...
Company operations: Thermo Fisher raises 2026 revenue guidance after Q1 strength
Thermo Fisher Scientific raised its 2026 revenue and adjusted EPS guidance after reporting Q1 2026 revenue growth and improved outlook. Chairman and CEO Marc Casper said full-year 2026 revenue is...
Microbial drug discovery: AI-designed antibiotic targets staph as resistance pressure persists
McMaster University researchers reported an AI generative model that explores 46 billion compounds and, in early tests, designed a brand-new antibiotic candidate targeting staphylococcal...